Humanized Mice at the Summer Toxicology Forum

The 43rd annual Summer Toxicology forum meeting was held on July 10-12, 2017 in Annapolis Maryland. The afternoon of July 10th was dedicated to a section titled "Use of Humanized Mice for Drug Development". The session was moderated by James Monroe, PhD, senior principle scientist at Merck Research Laboratories, and included presentations by researchers at the FDA, Merck, Yale, and Taconic Biosciences followed by a panel discussion featuring the presenters.

The first presentation was titled "Use of Humanized Mice to Analyze Human Biologics" presented by Kevan C. Herold, MD. Dr. Herold is a professor of Immunobiology and of Medicine (Endocrinology) at Yale University. He is also the executive director for the Yale Diabetes Center, and deputy director of the Yale Center for Clinical Investigation. Dr. Herold's presentation featured his work on oral administration of monoclonal antibodies in humanized mice. His research seeks to determine if route of administration can improve drug safety and potentiate tolerance to select biologics at key mucosal barriers1.

The second presentation was titled "Humanized Mouse Models: Progress and Perils" presented by Kristina Howard, DVM, PhD. Dr. Howard is the FDA Commissioner's Fellow, Therapeutic Proteins, Pharmaceutical Sciences, CDER, FDA, US FDA-CDER. Dr. Howard's presentation focused on her work on the use of humanized mouse models to better predict the biologic effects of therapeutic proteins in humans.

The next presentation was titled "Characterizing Humanized Immune System Mice for Use as Preclinical Safety Models" presented by Michael Oropallo, PhD, associate principle scientist at Merck. Dr. Oropallo's presentation featured a comprehensive head to head comparison of immune system reconstitution of first generation huNOG, and second generation huNOG-EXL mice.

The final talk in the presentation was titled "Scaling Production of Humanized Immune System Mice: Insights on the Consistency, Reproducibility, Strengths and Limitations of the huNOG and huNOG-EXL Humanized Mouse Models" presented by Michael Seiler, PhD. Dr. Seiler is the portfolio director for Commercial Genetically Engineered models at Taconic Biosciences, Inc. His presentation detailed the consistency and reproducibility of humanized mouse production at Taconic.

The session concluded with a panel discussion on the use and utility of humanized mice, and featured a series of questions from the audience. The three main messages resonating with attendees were:

The validity and recognition of data derived from humanized mouse studies by regulatory agencies.

The reliability and importance of vendor selection when purchasing humanized mice, especially next generation models.

The interest and intent of regulatory agencies and model providers to collaborate with pharma researchers.